A Research Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of DUR-928 in Patients With Alcoholic Hepatitis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Research Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of DUR-928 in Patients With Alcoholic Hepatitis (NCT03432260), a non-randomized phase 2 study. The primary outcome is Lille Model for Alcoholic Hepatitis Score.

2. Clinical Question

3. Trial Design

This completed Phase 2 study evaluated DUR-928 in patients with alcoholic hepatitis. It was open-label, non-randomized, and described as a parallel study. The registry lists 19 participants enrolled. The recorded start date was 2018-04-18, and the primary completion date was 2019-09-09.

The study description identifies dose-related safety, pharmacokinetics, and pharmacodynamics as its aims. The interventions were DUR-928 at 30 mg, 90 mg, and 150 mg. Arm descriptions distinguish patients with moderate AH from those with severe AH, and describe the doses as a dose-escalation series. The adverse-event listings include moderate-AH groups at 30 mg and 90 mg, and severe-AH groups at all three listed doses. The record does not explain the absence of a moderate-AH 150 mg group from those adverse-event listings.

Because assignment was not randomized and there was no listed control arm, comparisons among dose groups are descriptive rather than randomized treatment contrasts. Differences among groups could reflect factors other than dose, including differences in the patients enrolled. The registry record does not report the allocation procedure, the number assigned to each study arm for the efficacy endpoint, or a protocol-level explanation of how dose escalation proceeded. It also does not report treatment duration or the schedule of administration.

The primary endpoint was the Lille score on Day 7, summarized as a median and full range in the ITT population. The record lists 8 outcome measures and 0 analyses. Completion status indicates that the study is recorded as completed, but it does not establish that all planned analyses or outcome data were posted.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: ITT

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

6. Primary Endpoint

The registry lists the primary outcome as Lille Model for Alcoholic Hepatitis Score, measured over Day 7.

The primary endpoint is the Lille Model for Alcoholic Hepatitis Score, measured on Day 7. It is a calculated score rather than a direct measurement of a single laboratory value or a clinical event. Its inputs include age, serum albumin, creatinine, prothrombin time (PT), baseline bilirubin, and the difference between bilirubin before treatment and bilirubin on Day 7. The record specifies use of baseline values for all inputs except the Day 7 bilirubin value, with the screening value used when a Day 1 pre-dose value is unavailable.

The score ranges from 0.01 to 1.00. The registry description says a score >0.45 predicts a higher risk of death and recommends stopping steroid administration. The score is used in the context of predicting response to glucocorticoids, such as prednisolone, in AH. That context explains why the endpoint may be relevant to this population, but it does not make the score a direct measure of response to DUR-928.

The registry record specifies a median and full range at Day 7 for the ITT population. A median describes the middle of the observed values, while the full range describes the lowest and highest values. Neither the median nor the range is reported in the registry record, so the endpoint’s observed distribution cannot be described from this record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the primary summary describe?

The registry identifies the Lille score as a continuous score and specifies the median with the full range. General statistical explanation: the median is the value at the center of an ordered set of observations. It can describe a typical observation without being pulled as strongly by unusually high or low values as a mean may be. The full range gives the endpoints of the observed values, but does not show how many observations lie near either endpoint or how values are distributed between them. For that reason, a median and range provide a compact description rather than a complete picture of the data.

The registry record gives no actual median or range for the Lille score. It is therefore not possible to describe the observed score distribution, assess its variability, or compare distributions across the listed groups using the posted primary outcome information. The score’s definition and its stated range explain what the measurement represents, but they do not reveal what participants’ scores were.

Why does the study design matter for comparisons?

General statistical explanation: random allocation helps balance both measured and unmeasured participant characteristics across groups, on average. This study was non-randomized, so dose groups may differ in characteristics other than assigned dose. If outcomes differ between groups, a difference cannot automatically be attributed to dose. The record does not report enough detail to assess the allocation process or adjust for possible differences between groups.

The study is described as open-label, meaning masking was none. General statistical explanation: when treatment assignment is known, expectations or decisions about assessment and care can influence some recorded outcomes. This is a design consideration, not proof that such influence occurred in this study. The endpoint is a formula-based score using laboratory and clinical measurements, but the registry record does not describe procedures used to limit measurement or assessment bias.

The listed dose groups also include different AH severity categories. Moderate and severe AH are not interchangeable descriptions of the study population. A comparison that crosses severity groups could reflect severity as well as dose. The record does not report a planned statistical contrast that would account for this, and it does not identify a randomized control comparison. For these reasons, the dose listings should not be read as if they were equivalent arms of a controlled efficacy trial.

What can uncertainty measures tell a reader?

General statistical explanation: a confidence interval gives a range of values compatible with an estimate under the assumptions of the statistical method. Its width conveys precision: wider intervals indicate less precision, while narrower intervals indicate greater precision. A confidence interval does not eliminate uncertainty about study design, measurement, or bias. No confidence intervals for the primary outcome are reported in the registry record, so the precision of any primary-outcome estimate cannot be assessed.

A p-value, when reported, describes how compatible data are with a specified statistical model and null hypothesis. It is not a measure of the size or clinical importance of an effect. The registry record lists 0 analyses and does not report primary-outcome p-values. No inferential conclusion should be inferred from the fact that analyses are not listed.

How should the planning illustrations be read?

General statistical explanation: sample-size calculations can illustrate how many participants might be needed to detect a specified effect under a set of assumptions. The planning description identifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect defined as a fraction of the observed effect. It distinguishes participants per arm for non-time-to-event planning from total events for time-to-event planning. Such calculations are planning illustrations, not observed study results and not observed power.

The registry record does not report the trial’s own sample-size justification. The posted material does not give the effect estimates or planning results needed to interpret particular sample-size scenarios. The stated assumptions do not establish that the study was designed around those calculations, nor do they replace a protocol-level design justification. With no posted primary-outcome estimate, a reader cannot determine from the record how precisely the Lille score was measured.

Why does over-dispersion matter?

General statistical explanation: over-dispersion occurs when observed variability is greater than a statistical model expects. If a model assumes less variability than the data actually have, standard errors and uncertainty estimates may be too small. The registry record does not report a model for the primary outcome or evidence about over-dispersion. Since the primary endpoint is specified as a median and full range, the record does not establish that a model-based analysis was used. This concept is therefore relevant as a general consideration, not a finding about this study.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists 8 outcome measures and 0 analyses. The primary endpoint is the Day 7 Lille score, while the named secondary outcomes include serum Cytokeratin 18 (M30), serum Cytokeratin 18 (M65), percent change from baseline in INR, and percent change from baseline in bilirubin. The record does not describe how many statistical tests were planned or performed, or whether any adjustment for multiple comparisons was planned. A multiplicity strategy is therefore not reported in the registry record.

General statistical explanation: when researchers test multiple outcomes or contrasts, the chance of obtaining a seemingly notable result by chance can increase. Methods that account for multiple testing may be used, depending on the study’s objectives and analysis plan. The existence of several listed outcomes does not establish which tests were conducted, whether they were confirmatory, or whether adjustment was appropriate. The record does not provide enough information to assess those choices.

The primary outcome population is labeled ITT, but the registry record does not define how participants were included in that population or report its size. It does not state how missing Day 7 measurements were handled, whether participants with incomplete measurements were excluded, or whether any imputation method was used. The amount and pattern of missing outcome data are also not reported in the registry record. These omissions limit interpretation of the planned population and of any potential estimate.

The record does not report a statistical analysis plan, the handling of protocol deviations, or whether outcomes were analyzed according to assigned dose or treatment received. These details should not be inferred from the ITT label alone. Without reported methods and results, it is not possible to judge whether missing data or multiple outcomes influenced the conclusions of the study.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: anemia (25.0%), abdominal pain (25.0%), ascites (25.0%), hematemesis (25.0%), fluid overload (25.0%).

The registry reports serious adverse events by listed study group, including the number of participants at risk and the number affected. Serious adverse events were reported for 2 of 4 participants in the moderate-AH DUR-928 30 mg group, 0 of 3 in the moderate-AH DUR-928 90 mg group, 2 of 4 in the severe-AH DUR-928 30 mg group, 1 of 4 in the severe-AH DUR-928 90 mg group, and 0 of 4 in the severe-AH DUR-928 150 mg group. The record reports 0 deaths in each listed group.

Other adverse events were reported for 4 of 4 participants in the moderate-AH DUR-928 30 mg group, 2 of 3 in the moderate-AH DUR-928 90 mg group, 3 of 4 in the severe-AH DUR-928 30 mg group, 3 of 4 in the severe-AH DUR-928 90 mg group, and 3 of 4 in the severe-AH DUR-928 150 mg group. These counts describe participants with events; they do not give the total number of events per participant.

The listed serious-event terms—anemia, abdominal pain, ascites, hematemesis, fluid overload, and hypokalemia—are each reported at 25.0%. The denominator for those term percentages is not reported in the registry record. These small, non-randomized group counts are descriptive. They do not establish whether events were caused by DUR-928 or allow a reliable causal comparison among doses.

12. What the Result Does, and Does Not, Mean

The registry record establishes that a Phase 2 study assessed DUR-928 in patients with moderate or severe AH, using a non-randomized, open-label, dose-escalation design. It identifies the Day 7 Lille score as the primary endpoint and posts group-level safety counts. Those details describe what the study set out to assess and what safety information appears in the record.

The record does not report the primary endpoint values. There is no posted median or full range for the Lille score, nor are primary-outcome estimates or confidence intervals reported. It is not possible to say from this record whether scores changed, how they varied, or whether any dose group had a different score distribution. Missing endpoint results should not be interpreted as a finding of no effect.

The posted adverse-event counts provide a limited descriptive account. They do not establish causation, and they do not support a controlled comparison because allocation was non-randomized. Small group sizes also mean that counts can shift substantially with a small change in the number of participants affected. The record does not provide enough information to evaluate all potential safety outcomes or to characterize event timing and severity beyond the serious-event classification and posted counts.

The study’s scope includes pharmacokinetics and pharmacodynamics, but the registry record does not report results for those aims. It also does not report the study’s statistical analysis plan, handling of missing data, or multiplicity approach. Those omissions constrain interpretation beyond the primary outcome’s missing values.

Accordingly, the posted record supports a careful description of the design and the listed adverse events, but not a conclusion about the size, precision, or direction of an effect on the Lille score. It also does not establish comparative benefit or harm across doses.

13. Why This Trial Matters Statistically

This study is statistically informative as an example of the distinction between a clearly named endpoint and an interpretable result. The registry identifies the Lille score, its Day 7 measurement time, the summary statistic, and the named population. Yet the primary values are not reported. Without those values, readers cannot assess the observed distribution or the uncertainty around any comparison.

The design also matters. Non-randomized allocation and open-label assessment limit the strength of causal comparisons among dose groups. The groups are not a randomized control comparison, and the record does not describe methods that would make them comparable. This is especially important when the listed arms include both moderate and severe AH, since population severity may be relevant to outcomes.

The record’s safety counts illustrate why denominators and context matter. Counts are given for small groups, but they do not establish causation or a comparative risk. The serious-event terms are reported as percentages without a denominator identified in the registry record. Readers should distinguish these descriptive summaries from estimates designed to quantify a treatment effect.

Finally, the listed outcomes raise questions about analysis transparency. The record names multiple outcome measures but lists 0 analyses, and it does not report multiplicity or missing-data methods. These are not evidence of a flaw by themselves; they are gaps that limit what can be evaluated from the registry record. Clear outcome values, analysis methods, and uncertainty estimates are needed to connect a study’s question to a statistically interpretable result.

14. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

A central limitation of the registry record is that it does not report the primary endpoint results. Although the Lille score is defined and its summary method is specified, the median and full range are not posted. The record therefore does not permit direct interpretation of the primary outcome or its variability.

The design was non-randomized and open-label, with no listed control arm. Comparisons between dose groups are vulnerable to differences in participant characteristics and other factors beyond dose. The record does not report an allocation procedure, statistical adjustment strategy, or analysis plan that would clarify how such differences were addressed.

Enrollment was 19 overall, and the listed safety groups contain small numbers of participants at risk. Safety counts should therefore be interpreted descriptively. The registry does not report event-level timing, duration, or a causal assessment. It also does not specify the denominator for the listed serious-event term percentages.

The registry identifies an ITT population for the primary endpoint but does not define it or provide its size. Missing outcome data, imputation, protocol deviations, and the handling of participants who did not complete Day 7 assessment are not reported in the registry record. The record also does not describe adjustment for multiple outcomes or contrasts, despite listing 8 outcome measures and 0 analyses.

Finally, while pharmacokinetics and pharmacodynamics are study aims, corresponding results are not reported in the registry record. The trial’s sample-size justification and detailed dosing schedule are also not reported. These gaps limit assessment of both the statistical evidence and the study’s broader objectives.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: A Research Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of DUR-928 in Patients With Alcoholic Hepatitis — NCT03432260.
• Registry record last updated 2024-09-24; status last verified 2022-11.
• Results first posted 2022-12-14.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

18. Record Summary

NCT03432260 is a completed Phase 2, open-label, non-randomized, parallel study of DUR-928 in alcoholic hepatitis. The registry lists 19 participants enrolled and dose groups involving 30 mg, 90 mg, and 150 mg, with descriptions covering moderate and severe AH. The study aimed to assess safety, pharmacokinetics, and pharmacodynamic signals.

The primary endpoint was the Day 7 Lille score, summarized as a median and full range in the ITT population. The registry record does not report the primary endpoint values. It lists 8 outcome measures and 0 analyses. Group-level serious and other adverse-event counts are posted, along with serious-event terms, but the non-randomized design does not support a like-for-like controlled comparison. The record does not report the statistical analysis plan, missing-data methods, or multiplicity approach.